Molecule Promising Against Huntington's Disease

TUESDAY, Oct. 9 -- U.S. researchers say they've found a compound that may lead to a treatment for the hereditary, progressive neurological disorder known as Huntington's disease (HD).

In mice, a molecule called C2-8 appears to reduce HD-related neurological damage and delay the loss of motor control, said the team from the MassGeneral Institute for Neurological Disorders (MIND), Boston. Their study appears in the journal Proceedings of the National Academy of Sciences.

"We found that C2-8 slows the progress of HD in a mouse model and might do the same thing in human patients, if it or its biochemical relatives can be translated into a drug," study leader Dr. Steven Hersch said in a prepared statement. "What we don't know yet is precisely how it works, what molecules it interacts with in cells and how potent it might be."

His team first confirmed that oral doses of C2-8 can cross the blood-brain barrier and are nontoxic in mice. They also found that C2-8 doesn't interact with a number of molecules predictive of negative side effects.

HD mice that started receiving C2-8 when they were 24 days old did much better on tests of strength, endurance and coordination than HD mice that did not receive C2-8. The treated mice had less HD-related brain cell shrinkage and smaller aggregates of disease-related protein than untreated mice, the Boston group found.

The results suggest that C2-8 "has potential as the basis of a neuroprotective treatment," said Hersch, who is also an associate professor of neurology at Harvard Medical School. "We now need to confirm those results in a different mouse model, see whether similar compounds may be more potent than C2-8 and search for the enzyme or receptor that it is binding to."